JP6574383B2 - PCSK9iRNA組成物及びその使用方法 - Google Patents
PCSK9iRNA組成物及びその使用方法 Download PDFInfo
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- JP6574383B2 JP6574383B2 JP2015545846A JP2015545846A JP6574383B2 JP 6574383 B2 JP6574383 B2 JP 6574383B2 JP 2015545846 A JP2015545846 A JP 2015545846A JP 2015545846 A JP2015545846 A JP 2015545846A JP 6574383 B2 JP6574383 B2 JP 6574383B2
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- C—CHEMISTRY; METALLURGY
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- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
- C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
- A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/06—Antihyperlipidemics
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/21—Serine endopeptidases (3.4.21)
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/312—Phosphonates
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
- C12N2310/3533—Halogen
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen
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- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biotechnology (AREA)
- Epidemiology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Saccharide Compounds (AREA)
Applications Claiming Priority (9)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261733518P | 2012-12-05 | 2012-12-05 | |
| US61/733,518 | 2012-12-05 | ||
| US201361793530P | 2013-03-15 | 2013-03-15 | |
| US61/793,530 | 2013-03-15 | ||
| US201361886916P | 2013-10-04 | 2013-10-04 | |
| US61/886,916 | 2013-10-04 | ||
| US201361892188P | 2013-10-17 | 2013-10-17 | |
| US61/892,188 | 2013-10-17 | ||
| PCT/US2013/073349 WO2014089313A1 (en) | 2012-12-05 | 2013-12-05 | PCSK9 iRNA COMPOSITIONS AND METHODS OF USE THEREOF |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019016678A Division JP7239335B2 (ja) | 2012-12-05 | 2019-02-01 | PCSK9 iRNA組成物及びその使用方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2016506240A JP2016506240A (ja) | 2016-03-03 |
| JP2016506240A5 JP2016506240A5 (enExample) | 2017-01-19 |
| JP6574383B2 true JP6574383B2 (ja) | 2019-09-11 |
Family
ID=49841845
Family Applications (5)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015545846A Active JP6574383B2 (ja) | 2012-12-05 | 2013-12-05 | PCSK9iRNA組成物及びその使用方法 |
| JP2019016678A Active JP7239335B2 (ja) | 2012-12-05 | 2019-02-01 | PCSK9 iRNA組成物及びその使用方法 |
| JP2021025455A Withdrawn JP2021097680A (ja) | 2012-12-05 | 2021-02-19 | PCSK9 iRNA組成物及びその使用方法 |
| JP2023068284A Active JP7670747B2 (ja) | 2012-12-05 | 2023-04-19 | PCSK9 iRNA組成物及びその使用方法 |
| JP2025067855A Pending JP2025128073A (ja) | 2012-12-05 | 2025-04-17 | PCSK9 iRNA組成物及びその使用方法 |
Family Applications After (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019016678A Active JP7239335B2 (ja) | 2012-12-05 | 2019-02-01 | PCSK9 iRNA組成物及びその使用方法 |
| JP2021025455A Withdrawn JP2021097680A (ja) | 2012-12-05 | 2021-02-19 | PCSK9 iRNA組成物及びその使用方法 |
| JP2023068284A Active JP7670747B2 (ja) | 2012-12-05 | 2023-04-19 | PCSK9 iRNA組成物及びその使用方法 |
| JP2025067855A Pending JP2025128073A (ja) | 2012-12-05 | 2025-04-17 | PCSK9 iRNA組成物及びその使用方法 |
Country Status (30)
| Country | Link |
|---|---|
| US (2) | US10125369B2 (enExample) |
| EP (3) | EP2929031B9 (enExample) |
| JP (5) | JP6574383B2 (enExample) |
| KR (2) | KR102096014B1 (enExample) |
| CN (2) | CN104854242B (enExample) |
| AR (1) | AR093835A1 (enExample) |
| AU (4) | AU2013355237C1 (enExample) |
| BR (1) | BR112015013105B1 (enExample) |
| CA (1) | CA2892160C (enExample) |
| CY (2) | CY1120195T1 (enExample) |
| DK (1) | DK2929031T5 (enExample) |
| ES (1) | ES2657608T3 (enExample) |
| FR (1) | FR21C1021I2 (enExample) |
| HK (1) | HK1256621A1 (enExample) |
| HR (1) | HRP20180126T2 (enExample) |
| HU (2) | HUE035887T2 (enExample) |
| IL (2) | IL238917B (enExample) |
| LT (2) | LT2929031T (enExample) |
| LU (1) | LUC00209I2 (enExample) |
| MX (2) | MX367076B (enExample) |
| NL (1) | NL301107I2 (enExample) |
| NO (1) | NO2021024I1 (enExample) |
| NZ (1) | NZ709013A (enExample) |
| PL (1) | PL2929031T4 (enExample) |
| PT (1) | PT2929031T (enExample) |
| RS (1) | RS56783B9 (enExample) |
| SI (1) | SI2929031T1 (enExample) |
| SM (1) | SMT201800039T1 (enExample) |
| WO (1) | WO2014089313A1 (enExample) |
| ZA (1) | ZA201503829B (enExample) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2019103501A (ja) * | 2012-12-05 | 2019-06-27 | アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. | PCSK9 iRNA組成物及びその使用方法 |
Families Citing this family (65)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN104105790A (zh) * | 2011-11-18 | 2014-10-15 | 阿尔尼拉姆医药品有限公司 | 修饰的RNAi试剂 |
| EP3019200B1 (en) * | 2013-07-11 | 2022-03-23 | Alnylam Pharmaceuticals, Inc. | Oligonucleotide-ligand conjugates and process for their preparation |
| IL316808A (en) * | 2014-08-20 | 2025-01-01 | Alnylam Pharmaceuticals Inc | Modified double-stranded RNA materials and their uses |
| JOP20200115A1 (ar) * | 2014-10-10 | 2017-06-16 | Alnylam Pharmaceuticals Inc | تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات)) |
| WO2016057693A1 (en) * | 2014-10-10 | 2016-04-14 | Alnylam Pharmaceuticals, Inc. | Methods and compositions for inhalation delivery of conjugated oligonucleotide |
| JP6892433B2 (ja) | 2015-04-03 | 2021-06-23 | ユニバーシティ・オブ・マサチューセッツUniversity Of Massachusetts | 十分に安定化された非対称sirna |
| CA2995110A1 (en) | 2015-08-14 | 2017-02-23 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| CA2996701C (en) * | 2015-08-25 | 2024-05-28 | Alnylam Pharmaceuticals, Inc. | Methods and compositions for treating a proprotein convertase subtilisin kexin (pcsk9) gene-associated disorder |
| AU2017210726B2 (en) | 2016-01-31 | 2023-08-03 | University Of Massachusetts | Branched oligonucleotides |
| US12285464B2 (en) | 2016-04-18 | 2025-04-29 | The Trustees Of Columbia University In The City Of New York | Therapeutic targets involved in the progression of nonalcoholic steatohepatitis (NASH) |
| WO2018031933A2 (en) | 2016-08-12 | 2018-02-15 | University Of Massachusetts | Conjugated oligonucleotides |
| KR20240067272A (ko) * | 2016-10-18 | 2024-05-16 | 노파르티스 아게 | 전구 단백질 전환효소 섭틸리신 켁신 9 (pcsk9) 단백질 감소를 통한 심혈관 사건의 예방 방법 |
| FR3058061A1 (fr) * | 2016-10-27 | 2018-05-04 | Selexel | Nouvelle utilisation d'oligonucleotides double brin |
| CN108265052B (zh) * | 2016-12-30 | 2021-05-28 | 苏州瑞博生物技术股份有限公司 | 一种小干扰核酸和药物组合物及其用途 |
| CN110446785B (zh) | 2017-01-23 | 2024-04-26 | 瑞泽恩制药公司 | Hsd17b13变体及其应用 |
| JOP20190215A1 (ar) * | 2017-03-24 | 2019-09-19 | Ionis Pharmaceuticals Inc | مُعدّلات التعبير الوراثي عن pcsk9 |
| EP3607069B1 (en) | 2017-04-05 | 2022-11-02 | Silence Therapeutics GmbH | Products and compositions |
| KR20190139869A (ko) | 2017-04-11 | 2019-12-18 | 리제너론 파마슈티칼스 인코포레이티드 | 하이드록시스테로이드 (17-베타) 탈수소효소(hsd17b) 패밀리의 구성원의 조절인자의 활성도를 스크리닝하기 위한 검정 |
| EP3609532A1 (en) | 2017-04-13 | 2020-02-19 | Cadila Healthcare Limited | Novel peptide based pcsk9 vaccine |
| JP7406793B2 (ja) | 2017-06-23 | 2023-12-28 | ユニバーシティー オブ マサチューセッツ | 2テイル自己デリバリー型siRNAおよび関連方法 |
| US11597744B2 (en) | 2017-06-30 | 2023-03-07 | Sirius Therapeutics, Inc. | Chiral phosphoramidite auxiliaries and methods of their use |
| US10961583B2 (en) | 2017-10-11 | 2021-03-30 | Regeneron Phramaceuticals, Inc. | Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation |
| CN118291457A (zh) | 2017-12-01 | 2024-07-05 | 苏州瑞博生物技术股份有限公司 | 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途 |
| WO2019105435A1 (zh) | 2017-12-01 | 2019-06-06 | 苏州瑞博生物技术有限公司 | 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途 |
| WO2019105419A1 (zh) | 2017-12-01 | 2019-06-06 | 苏州瑞博生物技术有限公司 | 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途 |
| HRP20250277T1 (hr) | 2017-12-01 | 2025-05-09 | Suzhou Ribo Life Science Co., Ltd. | Dvolančani oligonukleotid, sastav i konjugat koji sadrži dvolančani oligonukleotid, postupak njihove pripreme i upotreba |
| US12083142B2 (en) | 2017-12-01 | 2024-09-10 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, composition and conjugate comprising the same, and preparation method and use thereof |
| EP3677679A4 (en) * | 2017-12-26 | 2020-12-02 | Guangzhou Ribobio Co., Ltd. | ARNSI MOLECULE INHIBITING PCSK9 GENE EXPRESSION AND ITS APPLICATION |
| CN109957565B (zh) * | 2017-12-26 | 2023-04-07 | 广州市锐博生物科技有限公司 | 一种修饰的siRNA分子及其应用 |
| CA3087106A1 (en) | 2017-12-29 | 2019-07-04 | Suzhou Ribo Life Science Co., Ltd. | Conjugates and preparation and use thereof |
| CA3091146A1 (en) | 2018-03-21 | 2019-09-26 | Regeneron Pharmaceuticals, Inc. | 17.beta.-hydroxysteroid dehydrogenase type 13 (hsd17b13) irna compositions and methods of use thereof |
| BR112020022546A8 (pt) * | 2018-05-07 | 2022-05-17 | Alnylam Pharmaceuticals Inc | Entrega extra-hepática |
| WO2020038377A1 (zh) | 2018-08-21 | 2020-02-27 | 苏州瑞博生物技术有限公司 | 一种核酸、含有该核酸的药物组合物和缀合物及其用途 |
| US11279930B2 (en) | 2018-08-23 | 2022-03-22 | University Of Massachusetts | O-methyl rich fully stabilized oligonucleotides |
| US11896674B2 (en) | 2018-09-30 | 2024-02-13 | Suzhou Ribo Life Science Co., Ltd. | SiRNA conjugate, preparation method therefor and use thereof |
| CN113454222B (zh) * | 2018-12-19 | 2025-01-14 | 阿尼拉制药公司 | 淀粉样前体蛋白(APP)RNAi剂组成物及其使用方法 |
| US11492619B2 (en) | 2019-01-18 | 2022-11-08 | University Of Massachusetts | Dynamic pharmacokinetic-modifying anchors |
| CA3141372A1 (en) * | 2019-05-22 | 2020-11-26 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use |
| BR112022002307A2 (pt) | 2019-08-09 | 2022-06-28 | Univ Massachusetts | Oligonucleotídeos quimicamente modificados que têm como alvo snps |
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Cited By (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2019103501A (ja) * | 2012-12-05 | 2019-06-27 | アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. | PCSK9 iRNA組成物及びその使用方法 |
| JP2021097680A (ja) * | 2012-12-05 | 2021-07-01 | アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. | PCSK9 iRNA組成物及びその使用方法 |
| JP7239335B2 (ja) | 2012-12-05 | 2023-03-14 | アルナイラム ファーマシューティカルズ, インコーポレイテッド | PCSK9 iRNA組成物及びその使用方法 |
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